EXPANDED ACCESS PROGRAMS

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1 EXPANDED ACCESS PROGRAMS PRACTICAL OPERATIONAL CONSIDERATIONS While patient demand for expanded access is growing, a clear consensus has yet to emerge on the best way to address this demand. Debate continues on the proper roles that the industry, FDA, treating physicians, patient advocates, and others should play. While there are no legal requirements for the industry to respond to direct requests for expanded access, patient advocacy groups continue to press for greater access and have successfully lobbied some states to adopt so-called right-totry laws. However, valid concerns have been raised that developing the programs necessary to provide expanded access potentially diverts time, money, energy, and resources away from the industry s core responsibility to deliver therapies faster to the market. Despite this chaotic landscape, it is important for biopharma companies to tackle the issues associated with expanded access thoughtfully and holistically by sharing their ideas and experiences while being transparent about the lessons learned. Janssen, in partnership with NYU s Division of Medical Ethics, recently published in JAMA a report of its experiences going through this process. 2 Beyond the operational lessons learned, these experiences are particularly relevant as they highlight the challenges an expanded access program faces in crafting solutions to complex ethical issues that involve questions of fundamental fairness, individual vs. greater good, etc. With increasing demand, growing public pressure, uncertain federal and state legislative status, and increased risks of negative social publicity, biopharma companies should proactively craft workable solutions to minimize the chance of impractical mandates being imposed on them. "EXPANDED ACCESS, SOMETIMES CALLED COMPASSIONATE USE OR PRE-APPROVAL ACCESS (PAA), IS THE USE OUTSIDE OF A CLINICAL TRIAL OF AN INVESTIGATIONAL MEDICAL PRODUCT FOR THE DIAGNOSIS, MONITORING, OR TREATMENT OF A SERIOUS DISEASE OR CONDITION WHEN THERE IS NO COMPARABLE OR SATISFACTORY ALTERNATIVE THERAPY AVAILABLE. 1 " FOOD AND DRUG ADMINISTRATION 1 Expanded Access (Compassionate Use) U.S. Food and Drug Administration Updated February 19, Accessed February 19, Caplan, A., Ray A. The Ethical Challenges of Compassionate Use. JAMA. doi: /jama Accessed February 17, 2016.

2 In formulating a holistic approach, there are a number of fundamental considerations: The need to offer expanded access programs at all The fiscal requirements to support these programs The right level of regulatory oversight The ability to address media pressures The relationship with patient advocate groups The medical and ethical implications The false hope these programs could inadvertently create As the larger ethical and policy issues continue to be debated, many biopharma companies continue to provide some measure of expanded access to certain therapies in their clinical pipeline. While many biopharma companies are very experienced at running global clinical trials, global expanded access programs pose special internal challenges. Based upon our extensive experience helping to set up and operate similar programs, we offer several recommendations for starting, scaling, and operating expanded access programs in your organization. INTERNAL CHALLENGES AND BEST PRACTICES While there are similarities between the decisions that must be made related to clinical development and expanded access, the latter is fraught with ethical and long-term nuanced issues. These should be thought through up front, whether developing a basic policy, or considering how to expand a program through post-trial and post-approval use. SUCCESSFUL EXPANDED ACCESS PROGRAMS REQUIRE THAT CAPABILITIES BE ESTABLISHED ACROSS MULTIPLE AREAS OF COMPETENCY WHILE DRAWING ON DIVERSE EXPERTISE ACROSS THE ORGANIZATION. Successful expanded access programs require that capabilities be established across multiple areas of competency while drawing on diverse expertise across the organization. These topic areas include, but are not limited to: Policy and scope Roles, responsibilities, and resourcing Regulatory compliance and reporting Communications, public relations Drug supply and logistics Data management Project management and training 2 North Highland Life Sciences Perspective

3 TOPIC AREA BEST PRACTICE Policy: Create policies to guide decisions on whether or not to make a particular drug available and through which expanded access program (e.g., early access programs, named patient programs, compassionate use programs). Determine the type and number of compounds that will be included and whether intermediate/large cohort programs and/or single patient programs will be implemented. Determine patient inclusion/exclusion criteria (e.g. treat only certain disease conditions). Define how to engage with various stakeholders (e.g., patients, physicians, sites, IRBs, advocacy groups, ethical review committees, regulatory groups). POLICY AND SCOPE NOTE: One of the more common mistakes we see is the development of policies that only respond to immediate requests and don t consider future indications that may be sought, implications for additional compounds in development, how early in the clinical development program single patient requests will be considered, what occurs post-approval, and what to do when there is no clear regulatory pathway in a particular geographic region or country. Scope: Create an integrated roadmap that takes into consideration: Impacts of planned commercial launch activities Transition to named patient/early access programs outside the U.S. Additional compound work Limits on the number of requests that will be considered The potential impact on clinical development and speed to market ROLES, RESPONSIBILITIES, RESOURCING REGULATORY COMPLIANCE & REPORTING The number of countries to be supported Give careful consideration to the number of resources required to operate an effective program vs. the degree to which those resources are needed to support ongoing and pending clinical trials. Pay particular attention to functions related to patient support, quality and safety, medical affairs, clinical trial management and operations, logistics/ supply, and communications/public relations. Align key stakeholders on program objectives. Assign clear roles and responsibilities for program management oversight. Determine resource needs to handle post-approval access for those with financial hardship, and in countries where approval has been granted but pricing has not been determined. Perform comprehensive assessment of compliance needs particularly for global programs and supplement central program staff with local resources (internal or external) as appropriate. 3 North Highland Life Sciences Perspective

4 Two topics are crucial to address: communications (internal/external) and training: External communications and public relations must be carefully controlled and well considered to manage the perceptions of numerous stakeholder groups with conflicting views (e.g., patient advocacy groups, regulators, legislators, ethicists). COMMUNICATIONS/ PUBLIC RELATIONS & TRAINING Internal communications serve to ensure that all employees, especially those in direct patient or physician contact, understand the company s policy, how to handle patient requests, and where to direct prospective patients, physicians or other interested external stakeholders for information. Given the sensitive nature of communications around these programs, don t underestimate the time and effort required to ensure alignment among legal, regulatory, public health, government, public affairs, medical affairs, medical operations, and other functions with specialized expertise. Developing sufficient training for anyone involved in the process is important so they understand not only the process, but also the context for the program and the expectations and impact of their role. Establish a specific point of contact within the clinical supply organization to manage and problem-solve drug supply issues. Conduct early forecast activities to determine allocation of supplies across clinical trial programs vs. early access programs. DRUG SUPPLY AND LOGISTICS Establish capability to meet unique needs of expanded access programs vs. clinical supply both inside and outside the U.S. (e.g., need for individually patient-labeled supply vs. general site labeling). Assess need and obtain external support for supply chain and logistics (in particular to address local country needs) and unique distribution requirements (e.g., whether or not the compound requires cold storage in transit). Determine how to measure and report on patient outcomes for these programs. DATA MANAGEMENT Make upfront decisions about what data to collect, track, and report on. Decisions about which patients to accept should be based on objective, sound clinical criteria that may come under scrutiny. Adding or modifying data while the program is in operation causes discrepancies, lost data, cross-functional miscommunication, and reporting difficulties. Establish standard domains, libraries, filing formats, etc. and enforce frequent quality checks of the data collection process and data quality itself. Determine data policies around obtaining and sharing patient information. Establish formal program management infrastructure, including communications, operational reporting and tracking, risk management, performance metrics, etc. PROGRAM MANAGEMENT Define end-to-end process from receipt of request to decision outcome and associated follow-ups. Identify all key impacted internal and external stakeholders and define a management plan. Provide formal status reporting to program sponsors. 4 North Highland Life Sciences Perspective

5 ACHIEVING OPERATIONAL EXCELLENCE Managing expanded access programs presents numerous operational challenges. But with careful consideration for the best practices outlined above, organizations can achieve the operational excellence needed to deliver effective solutions to all its stakeholders. ABOUT NORTH HIGHLAND North Highland is a global management consulting firm that delivers unique value, relevant big ideas, and strategic business capabilities to clients around the world. The firm solves complex business problems for clients in multiple industries through an integrated approach, and offers specialty services via its Data and Analytics, Managed Services, and Sparks Grove divisions. North Highland is an employee-owned firm that has been named as a Best Firm to Work For every year since 2007 by Consulting Magazine. The firm is a member of Cordence Worldwide ( a global management consulting alliance. For more information, visit northhighland.com and connect with us on LinkedIn, Twitter, and Facebook. For more information, please contact: Bob Pietrobono Vice President, Life Sciences Practice Lead bob.pietrobono@northhighland.com Jennifer Cahill Principal, Life Sciences jennifer.cahill@northhighland.com Peter Hess Principal, Life Sciences peter.hess@northhighland.com Copyright 2016 The North Highland Company. All Rights Reserved.